A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Public ClinicalTrials.gov record NCT00081497. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Multi-Center, Open-Label Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease That Previously Participated in the AGAL-008-00 Study
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. Fabrazyme (agalsidase beta) is a drug that helps to break down and removes certain types of fatty substances called "glycolipids". These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid (globatriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study analyzed the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease that previously participated in the AGAL-008-00 (NCT0074984) study.
Study identification
- NCT ID
- NCT00081497
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 4
- Enrollment
- 67 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 16 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 2004 (Type not available)
- Primary completion
- Sep 2005 (Actual)
- Completion
- Sep 2005 (Actual)
- Last update posted
- Apr 2, 2015
2004 – 2005
United States locations
- U.S. sites
- 18
- U.S. states
- 14
- U.S. cities
- 18
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294-0006 | — |
| Cedars-Sinai Medical Center | Los Angeles | California | 90048 | — |
| University of San Francisco | San Francisco | California | 94143 | — |
| University of Connecticut Health Partners | West Hartford | Connecticut | 06119 | — |
| Oncology Hematology Association | Coral Springs | Florida | 33065 | — |
| Emory University School of Medicine | Atlanta | Georgia | 30322 | — |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | — |
| University of Kansas Medical Center | Kansas City | Kansas | 66160-7233 | — |
| Gene Therapy Center - Department of Pediatrics and Institute of Human Genetics | Minneapolis | Minnesota | 55455 | — |
| Children's Hospital | Buffalo | New York | 14209 | — |
| Mount Sinai School of Medicine | New York | New York | 10029 | — |
| University of Rochester School of Medicine | Rochester | New York | 14642 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15261 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| University of Washington School of Medicine | Seattle | Washington | 98195 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 7 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00081497, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 2, 2015 · Synced Oct 18, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00081497 live on ClinicalTrials.gov.